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Results of First 36 Weeks of Treatment in Infants in Netherlands Published

Posted on: July 30, 2000

The Lancet—July 29, 2000

Recombinant human alpha-glucosidase from rabbit milk in Pompe patients
Lancet 2000; 356: 397-398
Hannerieke Van den Hout, Arnold J J Reuser, Arnold G Vulto, M Christa B Loonen, Adri Cromme-Dijkhuis, Ans T Van der Ploeg

Following is a short summary of the information published:

The results of the effects of the enzyme, human acid alpha-glucosidase, on four infantile Pompe patients was described by Ans Van der Ploeg, M.D, Ph.D., et al, in this issue of The Lancet Interactive. It documented the successful treatment of 4 babies with the infantile form of Pompe’s disease who were treated with recombinant human alpha-glucosidase from the milk of transgenic rabbits. The results showed that there was uptake of the enzyme in skeletal muscle and the muscle function was stimulated. The treatment reduced heart size, improved heart function and condition, and seemed to be responsible for the prolonged lives of the four babies in the clinical trial.

Pompe’s disease is a fatal muscle disease caused by the lack or deficiency of the lysosomal enzyme acid alpha-glucosidase (acid maltase). The infantile form of the disease is usually fatal within the first year of life. All four babies in the trial passed this critical one year juncture.

The report covered the first 36 weeks of the infantile clinical trial which began in January 1999, and is still ongoing in the Netherlands at Sophia Children’s Hospital, an affiliate of Erasmus University, Rotterdam.

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